D1.345 - Biologic Agents in Neurosarcoidosis: Efficacy and Future Perspectives from a Single-Center Cohort Study

Neurosarcoidosis is a rare manifestation of sarcoidosis, a systemic immune-mediated disease.  Given its rarity, there are no evidence-based treatment guidelines and the management depends on the severity of symptoms and the extent of neurological involvement, requiring a multidisciplinary approach to tailor treatment and optimize outcomes, while minimizing side effects. Biologic agents, particularly TNFα inhibitors, are increasingly used in refractory neurosarcoidosis failing to respond to first-line (steroids) or second line (classical DMARDs) treatments. This study aims to analyze a cohort of neurosarcoidosis patients treated with biologic agents.

We enrolled 49 consecutive neurosarcoidosis patients, followed at our Center. Data were collected retrospectively and prospectively to assess treatment outcomes.

Of 49 patients, 24 (49.0%) were treated with anti-TNFα agents (mean duration 32±30 months).  All of them were treated with steroids and DMARDs. Among these 24, biologics were used as first-line therapy in 3 patients (12.5%) and as third-line therapy in 87.5%. The introduction of anti-TNFα significantly reduced the number of disease relapses, from 2.44±1.5 relapses per patient before treatment to 0.12 after treatment, with a 95.1% reduction in the relapse rate.

Following the introduction of biologic therapy, 87.5% of patients remained relapse-free; only 3 patients (12.5%) experienced a single relapse each. In 2 of these 3, anti-infliximab antibodies were detected at the time of relapse.  Moreover, 19 patients (79.2%) were able to completely discontinue corticosteroid therapy after starting biologics. In addition, 4 patients (16.7%) successfully discontinued infliximab after achieving 3,5±1.3 years of disease stability and none of these 4 experienced a disease relapse 4,13±3 years of follow-up after discontinuation.

Anti-TNFα agents are effective in reducing disease relapses, enabling steroid reduction or withdrawal, and achieving long-term disease stability in patients with refractory disease.

Over time, we registered an increasing trend in the prescription of anti-TNFα agents (14/24 patients started treatment in the last 2 years) and a decreasing time of prescription since initial diagnosis. This underscores the growing role of biologics in the treatment of neurosarcoidosis. Looking forward, biologic therapies may play an even more central role in treatment strategies, with earlier prescription without waiting for patients to experience multiple relapses and steroid-related side effects.